Mitsubishi Tanabe Pharma America announced the initiation of a global Phase 3 study evaluating the long-term safety and tolerability of an investigational oral suspension formulation of edaravone (MT-1186) in patients with amyotrophic lateral sclerosis (ALS).
The intravenous (IV) formulation of edaravone, known as RADICAVA® (edaravone), is a treatment option previously approved by the U.S. Food and Drug Administration (FDA) that was shown in a prior clinical study to slow the loss of physical function in ALS patients.
"Enrollment of the first patient in this clinical study marks an important milestone for our company as we continue to seek treatment options for the ALS community," said Atsushi Fujimoto, President, MTPA. "There is a great unmet need in the area of ALS, and we believe an oral formulation of edaravone could potentially serve as an alternative treatment option for patients suffering from this progressive condition."
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The global, multi-center, open-label study, sponsored by Mitsubishi Tanabe Pharma Development America (MTDA), will evaluate 150 people with ALS across an estimated 65 sites in the U.S., Canada, Europe and Japan over the course of 48 weeks of treatment. Approximately half the study sites will be located in the U.S.
After a screening period of up to 21 days, study participants (18 to 75 years of age) will receive oral edaravone following the same dosing regimen currently utilized by patients on the IV formulation.
In the study, the primary safety assessments will include adverse events, physical examination, electrocardiogram parameters, vital signs, laboratory safety assessments, unsteadiness and sensory evaluations. Exploratory endpoints will include change in ALS Functional Rating Scale-Revised (ALSFRS-R) score, change in percentage of forced vital capacity (FVC), and time (days) to tracheostomy, or permanent assisted mechanical ventilation.
"ALS presents a tremendous burden to patients as the disease robs them of physical function," said Carlayne E. Jackson, M.D., a U.S. principal investigator. "As a researcher and provider of care for ALS patients, I'm pleased to be part of a research effort with a goal to provide a new treatment option for those living with this devastating disease."